-- Rett syndrome research to include oral
presentation of Phase 3 Lavender trial and poster presentation
highlighting the patient journey and clinical burden of the
disease
-- E-poster presentation evaluating maintenance
of efficacy and safety of pimavanserin among patients with
Parkinson’s disease dementia with psychosis
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced a
late-breaking oral presentation on the efficacy and safety data
from the Phase 3 Lavender study of trofinetide, an investigational
treatment for Rett syndrome (RTT), will be presented at the 2022
American Academy of Neurology Annual Meeting (AAN), held in
Seattle, WA, April 2-7, and virtually April 24-26, 2022.
“We look forward to the upcoming presentation of the Phase 3
Lavender results at this year’s AAN and the opportunity to educate
neurologists on the critical unmet need that exists for patients
and their caregivers living with this rare and serious neurological
disease,” said Kathie Bishop, Ph.D., Acadia’s Senior Vice
President, Chief Scientific Officer and Head of Rare Disease.
AAN Accepted Presentations and Posters:
Emerging Science (Late-Breaker):
- Oral Presentation: Efficacy and safety of trofinetide
for the treatment of Rett syndrome: results from the pivotal phase
3 Lavender study. Tuesday, April 5, from 9:15 a.m. -
11:30 a.m.
Posters:
- Poster # 001: Patient journey and clinical burden of
Rett syndrome in the United States: research study including nearly
6,000 girls provides an overview of the patient journey and
clinical burden of RTT with respect to baseline characteristics,
clinical manifestations and treatment patterns among female
individuals in the US. Tuesday, April 5, from 11:45
a.m. - 12:45 p.m.
- E-Poster # P1.006: Pimavanserin treatment of
hallucinations and delusions in patients with Parkinson’s disease
dementia: post hoc analysis of the Phase 3 HARMONY clinical trial.
Analysis evaluating the maintenance of efficacy, as measured by
time to relapse, of pimavanserin in a subgroup of patients with
Parkinson’s disease dementia with psychosis during the 26-week
maintenance phase of the HARMONY clinical trial. Virtual
American Academy of Neurology Annual Meeting, April 24-26.
About Rett Syndrome
Rett syndrome is a rare, debilitating neurological disorder that
occurs primarily in females following apparently normal development
for the first six months of life. Rett syndrome is often
misdiagnosed as autism, cerebral palsy, or non-specific
developmental delay. Rett syndrome is caused by mutations on the X
chromosome on a gene called MECP2. There are more than 200
different mutations found on the MECP2 gene that interfere with its
ability to generate a normal gene product. Rett syndrome occurs
worldwide in approximately one of every 10,000 to 15,000 female
births and in the United States impacts 6,000 to 9,000 patients.
Rett syndrome causes problems in brain function that are
responsible for cognitive, sensory, emotional, motor and autonomic
function. Typically, with symptoms presenting between six to 18
months of age, patients experience a period of rapid decline with
loss of purposeful hand use (fine motor skills), development of
hand stereotypies, absent or impaired mobility (gross motor
skills), loss of communication skills (including eye contact) and
inability to independently conduct activities of daily living.
Symptoms also include seizures, disorganized breathing patterns, an
abnormal side-to-side curvature of the spine (scoliosis), and sleep
disturbances. Currently, there are no FDA-approved medicines for
the treatment of Rett syndrome.
About Trofinetide
Trofinetide is an investigational drug. It is a novel synthetic
analog of the amino‐terminal tripeptide of IGF-1 designed to treat
the core symptoms of Rett syndrome by potentially reducing
neuroinflammation and supporting synaptic function. Trofinetide is
thought to stimulate synaptic maturation and overcome the synaptic
and neuronal immaturities that are characteristic of Rett syndrome
pathophysiology. In the central nervous system, IGF-1 is produced
by both of the major types of brain cells – neurons and glia. IGF-1
in the brain is critical for both normal development and for
response to injury and disease. Trofinetide has been shown to
inhibit the production of inflammatory cytokines, inhibit the
overactivation of microglia and astrocytes, and increase the amount
of available IGF-1 that can bind to IGF-1 receptors. Trofinetide
has been granted Fast Track Status and Orphan Drug Designation for
Rett syndrome and has also been granted Rare Pediatric Disease
(RPD) designation by the FDA.
About Parkinson’s Disease and Parkinson’s Disease Psychosis
Parkinson’s disease is a progressive nervous system disorder
that affects about one million people in the United States.1,2 The
signs and symptoms can vary with people experiencing both motor
symptoms and non-motor symptoms such as hallucinations (seeing,
hearing, or experiencing things that others don’t) and delusions
(false beliefs).3,4 Physicians may refer to these
Parkinson’s-related hallucinations and delusions as Parkinson’s
disease psychosis.4 Around 50 percent of people living with
Parkinson’s may experience hallucinations or delusions during the
course of their disease.3 Non-motor symptoms, as a whole, can be
more troublesome than motor symptoms, in terms of quality of life.5
Parkinson’s disease psychosis may add to the burden of caring for a
loved one with Parkinson’s disease.6,7
About Pimavanserin
Pimavanserin is a selective serotonin inverse agonist and
antagonist preferentially targeting 5-HT2A receptors. These
receptors are thought to play an important role in neuropsychiatric
disorders. In vitro, pimavanserin demonstrated no appreciable
binding affinity for dopamine (including D2), histamine,
muscarinic, or adrenergic receptors. Pimavanserin was approved for
the treatment of hallucinations and delusions associated with
Parkinson’s disease psychosis by the U.S. Food and Drug
Administration in April 2016 under the trade name NUPLAZID®.
NUPLAZID is not approved for Alzheimer’s disease psychosis. In
addition, Acadia is developing pimavanserin in other
neuropsychiatric conditions.
About Acadia Pharmaceuticals
Acadia is advancing breakthroughs in neuroscience to elevate
life. For more than 25 years we have been working at the forefront
of healthcare to bring vital solutions to people who need them
most. We developed and commercialized the first and only approved
therapy for hallucinations and delusions associated with
Parkinson’s disease psychosis. Our late-stage development efforts
are focused on treating psychosis in patients with dementia, the
negative symptoms of schizophrenia and Rett syndrome. Our
early-stage development efforts are focused on novel approaches to
pain management, cognition and neuropsychiatric symptoms in central
nervous system disorders. For more information, visit us at
www.acadia-pharm.com and follow us on LinkedIn and Twitter.
Important Safety Information and Indication for NUPLAZID®
(pimavanserin)
Indication
NUPLAZID is indicated for the treatment of hallucinations and
delusions associated with Parkinson’s disease psychosis.
Important Safety Information
WARNING: INCREASED MORTALITY IN ELDERLY PATIENTS WITH
DEMENTIA-RELATED PSYCHOSIS
- Elderly patients with dementia-related psychosis treated
with antipsychotic drugs are at an increased risk of
death.
- NUPLAZID is not approved for the treatment of patients with
dementia-related psychosis unrelated to the hallucinations and
delusions associated with Parkinson’s disease psychosis.
Contraindication: NUPLAZID is contraindicated in patients
with a history of a hypersensitivity reaction to pimavanserin or
any of its components. Rash, urticaria, and reactions consistent
with angioedema (e.g., tongue swelling, circumoral edema, throat
tightness, and dyspnea) have been reported.
Warnings and Precautions: QT Interval Prolongation
- NUPLAZID prolongs the QT interval. The use of NUPLAZID should
be avoided in patients with known QT prolongation or in combination
with other drugs known to prolong QT interval including Class 1A
antiarrhythmics or Class 3 antiarrhythmics, certain antipsychotic
medications, and certain antibiotics.
- NUPLAZID should also be avoided in patients with a history of
cardiac arrhythmias, as well as other circumstances that may
increase the risk of the occurrence of torsade de pointes and/or
sudden death, including symptomatic bradycardia, hypokalemia or
hypomagnesemia, and presence of congenital prolongation of the QT
interval.
Adverse Reactions: The common adverse reactions (≥2% for
NUPLAZID and greater than placebo) were peripheral edema (7% vs
2%), nausea (7% vs 4%), confusional state (6% vs 3%), hallucination
(5% vs 3%), constipation (4% vs 3%), and gait disturbance (2% vs
<1%).
Drug Interactions:
- Coadministration with strong CYP3A4 inhibitors (e.g.,
ketoconazole) increases NUPLAZID exposure. Reduce NUPLAZID dose to
10 mg taken orally as one tablet once daily.
- Coadministration with strong or moderate CYP3A4 inducers
reduces NUPLAZID exposure. Avoid concomitant use of strong or
moderate CYP3A4 inducers with NUPLAZID.
Dosage and Administration
Recommended dose: 34 mg capsule taken orally once daily, without
titration.
NUPLAZID is available as 34 mg capsules and 10 mg tablets.
Please read the full Prescribing Information including Boxed
WARNING.
Forward-Looking Statements
Statements in this press release that are not strictly
historical in nature are forward-looking statements. These
statements include but are not limited to statements regarding the
timing of future events. These statements are only predictions
based on current information and expectations and involve a number
of risks and uncertainties. Actual events or results may differ
materially from those projected in any of such statements due to
various factors, including the risks and uncertainties inherent in
drug development, approval and commercialization. For a discussion
of these and other factors, please refer to Acadia’s annual report
on Form 10-K for the year ended December 31, 2021 as well as
Acadia’s subsequent filings with the Securities and Exchange
Commission. You are cautioned not to place undue reliance on these
forward-looking statements, which speak only as of the date hereof.
This caution is made under the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995. All
forward-looking statements are qualified in their entirety by this
cautionary statement and Acadia undertakes no obligation to revise
or update this press release to reflect events or circumstances
after the date hereof, except as required by law.
References
1Parkinson’s Disease Foundation. What is Parkinson’s disease?
Retrieved from
https://www.parkinson.org/Understanding-Parkinsons/Statistics.
Accessed March 2021. 2Postuma RB, Berg D, Stern M, et al. MDS
clinical diagnostic criteria for Parkinson’s disease. Mov Disord.
2015;30(12):1591-1601. 3Forsaa EB, Larsen JP, Wentzel-Larsen T, et
al. A 12-year population-based study of psychosis in Parkinson’s
disease. Arch Neurol. 2010;67(8):996-1001. 4Ravina B, Marder K,
Fernandez HH, et al. Diagnostic criteria for psychosis in
Parkinson’s disease: report of an NINDS, NIMH work group. Mov
Disord. 2007;22(8):1061-1068. 5Martinez-Martin P,
Rodriguez-Blazquez C, et al. The Impact of Non-Motor Symptoms on
Health-Related Quality of Life of Patients with Parkinson’s
Disease. Mov Disord. 2011;26(3):399-406. 6Schrag A, Hovris A, et
al. Caregiver-burden in parkinson’s disease is closely associated
with psychiatric symptoms, falls, and disability. Parkinsonism and
Related Disorders. 2006;12:35-41. 7Aarsland D, Bronnick K, Ehrt U.
et al. Neuropsychiatric symptoms in patients with Parkinson’s
disease and dementia: frequency, profile and associated care giver
stress. J Neurol Neurosurg Psychiatry. 2007;78:36-42.
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version on businesswire.com: https://www.businesswire.com/news/home/20220322005339/en/
Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818)
395-3043 media@acadia-pharm.com
Investor Contact: Acadia Pharmaceuticals Inc. Mark Johnson, CFA
(858) 261-2771 ir@acadia-pharm.com
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